地夫可特的副作用与注意事项

Deflazacort is a corticosteroid drug for Duchenne muscular dystrophy (DMD), produced by PTC Therapeutics of the United States. Although it has shown significant efficacy in treating DMD, it also comes with a series of side effects and precautions. This article will discuss in detail the common side effects of deflazacort, precautions when using it, and its efficacy in the treatment of DMD, to help patients and caregivers better understand this drug.

Side effects of deflazacort

As a corticosteroid, deflazacort’s side effects involve multiple systems, and patients need to pay close attention to their body’s reactions during use. The following will be explained from three aspects: endocrine, immune and skeletal systems.

Effects on the endocrine system

Long-term use of deflazacort may lead to Cushing's syndrome, which is manifested by symptoms such as weight gain, round face, and high blood pressure. In addition, it may cause high blood sugar and even induce diabetes, and patients need to monitor blood sugar levels regularly. Sudden discontinuation of the drug may also cause adrenal insufficiency, manifested as fatigue, hypotension, etc. Therefore, the dose needs to be gradually reduced when the drug is discontinued.

Immune system suppression

Deflazacort can reduce immunity and increase the risk of infection, including bacterial, viral, or fungal infections. Patients should avoid contact with people with chickenpox or measles and be vaccinated if necessary. For hepatitis B virus carriers, drugs may activate viral replication and require early screening and monitoring.

Bone and muscle problems

Long-term use may lead to osteoporosis or femoral head necrosis, and children may also experience growth retardation. In addition, concomitant use of deflazacort with neuromuscular blocking agents may cause acute myopathy, which may manifest as muscle weakness or even paralysis.

Understanding these side effects can help patients identify abnormal reactions in time and adjust their medication regimen under the guidance of a doctor.

Precautions for deflazacort

In order to use deflazacort safely, patients need to follow a number of precautions, covering medication adjustments, special populations, and drug interactions.

Dose adjustment and monitoring

When used in combination with a CYP3A4 inhibitor, the dose of deflazacort needs to be reduced to one-third of the original dose. At the same time, patients should regularly check their blood pressure, blood sugar, and bone density to detect potential problems in a timely manner.

Use by special groups

Using by pregnant women may increase the risk of fetal malformations, and lactating women should be cautious. The safety of children under 2 years old has not yet been determined, and elderly patients should take the medication under the guidance of a doctor. Patients with hepatic and renal impairment usually do not require dose adjustment, but patients with severe hepatic impairment require individualized evaluation.

Drug-food interactions

Avoid combined use with strong CYP3A4 inducers (such as rifampicin) to avoid reducing drug efficacy. A high-fat diet may delay drug absorption but does not affect overall efficacy.

Strictly following these precautions can minimize the risks of medication and improve the safety of treatment.

The efficacy of deflazacort

Deflazacort has shown significant clinical value in the treatment of Duchenne muscular dystrophy (DMD). Its efficacy is mainly reflected in delaying disease progression and improving patients' quality of life.

Delay disease progression

Studies have shown that deflazacort can effectively slow down the degeneration of muscle function in DMD patients and extend the time of independent walking. Its anti-inflammatory and immunomodulatory effects help protect muscle tissue.

Improve quality of life

By reducing inflammatory responses, deflazacort can reduce muscle pain and stiffness, improve patients' mobility, and some patients may also improve their cardiopulmonary function.

Comparison with other drugs

Compared with other corticosteroids (such as prednisone), deflazacort has a slightly different side effect profile and may be better tolerated by some patients, but the specific choice needs to be determined based on individual circumstances.

Deflazacort provides an important treatment option for DMD patients. Rational use can control symptoms while reducing adverse reactions.